SANFILIPPO CHILDREN’S FOUNDATION - FUNDING RESEARCH, FINDING GEMS

Our vision: Fund medical research so a cure can be found in time for children battling Sanfilippo today and those born with it tomorrow INTRODUCTION - OUR YEAR IN REVIEW HIGHLIGHTS WE’VE ACHIEVED TOGETHER Welcome

• Partnership sealed with biotech, Abeona Therapeutics Inc As we conclude our second year of operation we are extremely encouraged in this (NASDAQ: ABEO ) to deliver clinical gene therapy trials time of change, expansion, philanthropic gestures and pioneering scientific progress. • Invested the first USD$200,000 with a total of USD$1m committed It’s been an extraordinary year for the Sanfilippo Children’s Foundation and we couldn’t (est. AUD$1.3m) towards first clinical research program

have done it without the help of our exceptional community of supporters. We thought • Australian clinical team engaged to establish trial site in we would show you some of what we have been up to. • Over 5,000 Facebook followers

• Facebook total reach peaks at 40,000 in December 2014

• Over 6,000 supporters on our database WELCOME AND HIGHLIGHTS 02 • Foundation ambassador and Aussie singers band together to OUR MAJOR CONTRIBUTORS 04 launch dedicated song

“HOPE FOR” CAMPAIGNS 06 • Extensive media coverage including 5 TV segments, 42 print OUR GEM FUNDRAISERS 08 articles and 12 radio interviews

GEM QUOTES 11 • 5 major corporations donate over $100,000 in gift matching alone

MEDIA COVERAGE 12 • Dedicated office space secured for the Foundation

SPECIAL THANKS 14 • 40 community events raising over $100,000 A MESSAGE FROM OUR FOUNDER 15 • 100 peer-to-peer fundraising campaigns raising in excess of THE TEAM 16 $90,000

AMBASSADORS 17 • New fully integrated, interactive and supporter friendly website FAMILY STORIES 18 launched

THE FINANCIALS 20 • Family-led “Hope for” campaigns combined raised more than THE RESEARCH 22 $140,000 • Welcomed 2 new ambassadors

• Opened an online store and launched a line of SCF merchandise. This Year in Review document has been produced by professionals who have discounted and/or donated their time and services. We would like to thank designer Alex Moller and Lindfield Print and Copy Centre.

2 Sanfilippo Children’s Foundation Our year in review - 2015 3 THANK YOU TO OUR GEM FUNDRAISERS

DICK SMITH FOODS

The Sanfilippo Children’s Foundation won $50,000 from the Dick Smith Foods Foundation in its $1 Million Charity Breakthrough campaign. It is a heart-warming story of people power with SCF supporters taking more ‘selfies’ and MAJOR CONTRIBUTORS casting more votes than over 450 other eligible Australian charities over a six-month period. The SCF took out top gong in an initiative philanthropist and business man Dick Smith has called the “ultimate crowdfunding”.

Sponsors and Events that helped raise funds in 2015

SANFILIPPO 1000 – BIG FEET FOR LITTLE FEET

Proudly sponsored by Westpac, a highly motivated team of 7 runners relayed from Melbourne to Sydney and brought home a staggering $166,000 for the Sanfilippo Children’s Foundation, raising immeasurable awareness across both regional and metropolitan Victoria and New South Wales whilst exceeding all fundraising targets.

A W EDWARDS

The Sanfilippo Children’s Foundation was nominated by David Oliver as the recipient of the A W Edwards Charity Shield Day, a barefoot bowls event held in North Sydney in October. Thanks to the extraordinary support of A W Edwards, event sponsors and suppliers, the SCF was presented with a cheque for $90,000.

KCA WALKATHON

In 2014 Kimberly-Clark Australia (KCA) staff participated in a company and nation-wide KCA Walkathon. So successful was the event and relationship with the SCF, that the KCA team hit the pavement again in 2015 to raise additional funds.

For two years running, KCA staff have participated in a series of fundraising events and activities. Thank you to HIDDEN GEMS GOLF DAY KCA employee Abby Phillips, who upon sharing the mission of the Foundation, has continued to rally the troops. With KCA matching, this year the event raised $82,500 towards our research programs, bringing the combined Due to the success of the inaugural event in 2014, this year golfers supported the SCF at the Hidden Gems total to $162,500 over the last 2 years. Golf Day at Cromer in Sydney. Golfers embraced the day with an Ambrose style event whilst partaking in wine tasting and competitions on course before retiring to the club for lunch and entertainment by ex-cricketer Kerry O’Keeffe. With thanks to our volunteer organiser, Stephanie Donohoe, the event raised $30,000.

4 Sanfilippo Children’s Foundation Our year in review - 2015 5 THANK YOU TO OUR GEM FUNDRAISERS

HOPE FOR KYUSS

March saw the launch of the Hope for Kyuss campaign, as the Harris and Johnson families rallied significant “HOPE FOR” CAMPAIGNS community support in regional Victoria. Along with two other children with Sanfilippo (Meckenzie Oelofsen and Skye Robson), Kyuss was featured in a Channel 9 news story about the plight of three families battling this devastating disease. The launch of the Hope for Kyuss campaign was also featured in the family’s local newspaper Successful family-led fundraising campaigns humanise the face of Sanfilippo Syndrome the Yarra Valley Leader and successful fundraising events have since included a ‘Sex Outta the City’ fundraiser, the ‘Ky Cup’ and the ‘Ky-T’ high tea fundraisers. Hope for Kyuss has raised over $93,000 this year.

HOPE FOR ISLA & JUDE

In December 2014, the Sanfilippo Children’s Foundation made national and international headlines with the launch of our Hope for Isla and Jude campaign and the release of the charity single This Woman’s Work. The cover of the Kate Bush song was produced by Foundation ambassador, Sébastien Izambard of Il Divo pop opera fame, together with Sony Music Australia.

Sébastien visited Sydney to produce the single and united a stellar list of Australian artists including Darren Hayes, Natalie Bassingthwaighte, Ella Hooper, Diesel, David Campbell, Alex Lloyd, Christine Anu, (Empire of the Sun), Katy Steele (), Casey Donovan, Damien Leith, Nathaniel, Marlisa, and the Australian Children’s Choir. HOPE FOR MECKENZIE The charity anthem was accompanied by a film clip produced by film-maker, Jason van Genderen from Treehouse Creative. The campaign generated over $50,000 in donations and associated fundraising activity and Meckenzie recently turned 8 and to launch her Hope for campaign, Meckenzie’s family announced she has 8 the film clip has been viewed over 58,000 times. The campaign generated significant media coverage including birthday wishes - 1. Life, 2. Hope, 3. Treatment, 4. Help, 5. Cure, 6. Awareness, 7. Donate and to 8. Grow Old! 4 TV segments (Sky News, Channel 9 Mornings, Channel 9 Today Show and Channel 7 Morning Show), 10 print Meckenzie has had three articles appear in her local paper, The Moorabbin Leader, and also appeared in the articles and 6 radio interviews. Channel 9 news story filed by journalist and Foundation ambassador Sonia Marinelli.

HOPE FOR SKYE HOPE FOR ALEC

In February 2015, we introduced Skye Robson with the launch of the Hope for Skye campaign with Skye’s family Alec Morrice has Sanfilippo Type C, a rarer form of Sanfilippo affecting only 1 in every 1.5 million live births. joining the fight to raise funds. Their first #BlueSkyforSkye social media campaign on Rare Disease Day (28 Feb) Alec was diagnosed at the beginning of 2015. On the front foot in the fight to raise awareness about this was a great success with good traction, and generated a series of grassroots events in Ballarat, Victoria, and also in devastating disease, Alec’s family has been quick to get on-board and all money raised by the Hope for Alec Thailand, where Skye also has family. The Hope for Skye campaign has been profiled in four articles in theBallarat campaign will be put towards advancing research into Sanfilippo Type C. So far this year, over $11,000 has Courier and twice in The Miner Newspaper. This year, the Hope for Skye campaign has raised almost $40,000. been raised by Alec’s supporters.

6 Sanfilippo Children’s Foundation Our year in review - 2015 7 THANK YOU TO OUR GEM FUNDRAISERS OUR GEM FUNDRAISERS

PUB 2 PUB FUN RUN In the spirit of “people-powered medicine”, BUNNING’S CAKE STALLS KY-T (HIGH TEA) FUNDRAISER MUAY THAI BOXING EVENT grassroots community support for the Sanfilippo two years running cause continues to grow and is paramount in realising our goals. In the last year, the SCF supported approximately 40 community events, raising over $100,000. Grassroot events have been held across the country by everyday people who we thank for their extraordinary support.

We’ve had runners, walkers, bakers, riders, dieters, singers, head and beard shavers and even bag-pipers. There have been many events including: trivia, discos, Bunnings BBQs, Scottish dancing, endorsed golf days, band and karaoke nights, car boot sales, book fairs, car park collections, online auctions, gala dinners, street buskers, Christmas parties, cake stalls and group fun runs and walks.

Peer-to-peer fundraising continues to grow with approximately 50 individuals fundraising for the SCF with a personalised fundraising page on the Foundation website. Fundraisers secured sponsorship via physical challenges including fun runs, marathons, obstacle courses and bike rides as well as challenges and dares such as giving up chocolate, wearing a wig for a month, and dying hair purple.

DANCE FOR HOPE VIOLET DIAMOND BALL, SUN RUN SYDNEY TRIVIA SEX OUTTA THE CITY OPEN GARDEN FUNDRAISER two years running MELBOURNE two years running two years running FUNDRAISER

8 Sanfilippo Children’s Foundation Our year in review - 2015 9 A few words from some of our THANK YOU TO OUR GEM FUNDRAISERS GEM QUOTES Gem fundraisers about their involvement in the Sanfilippo Children’s Foundation. PINT-SIZED PHILANTHROPISTS

Children are often the Sanfilippo Children’s Foundation’s most avid supporters, exercising John Burgman Belinda Yates philanthropy and a sense of community well ahead Westpac employee Bunnings cake stall of their years. Fundraisers led by children have and instigator of the organiser & regular included kerbside cake stalls, birthday celebrations Sanfilippo 1000 volunteer in lieu of gifts, busking, making Sydney Harbour Bridge-length loom bands and competing in fun runs. “I’m linked to SCF via Allan Donnell from playing “Our babies (Isla and Xavier) were a month old soccer for Manly Vale. It was absolutely heart- when Megan and I met at mother’s group. When the breaking to see a mate and his family deal with the Foundation was created I could see the drive Megan Sanfilippo diagnosis and impossible to comprehend had. Listening to her talk about this, well… it was hard there was so little hope initially offered. not to be inspired.

Despite the adversity, it’s been inspirational to see My experience with breast cancer had made me the courage, determination and passion from Meg realise how precious life is. I feel very grateful there is and Al (and the broader SCF community) on not only a treatment for breast cancer. It really made me want their quest for a cure but their selfless support of the same for Isla and Jude: a treatment, medicine… other Sanfilippo families.” and an opportunity for life!”

Peta Goodlet Rachel Hansen Event organiser Paediatric and Hope for Kyuss Occupational committee member Therapist at Kids OT

“I was inspired to help after attending the Violet Diamond Ball. I already knew Kyuss and Nikki’s story “Over the past 12 months, Kids OT has participated but left the Ball motivated having learnt more about in fun runs, painted faces, sold lucky dips and the disease and the urgent need for a treatment. Christmas decorations, got involved in the ‘Loom for Sanfilippo’ initiative and taken as many Dick Smith With no government funding or the resources of a selfies as possible. large charity, this was a cause that needed a little extra help. I couldn’t sit on my hands. A group of Not only do our individual team members gain joy girls formed the ‘Hope for Kyuss’ committee. We all from participating, but as a workplace, this has had bring something to the table and have a mix of skills, a hugely positive influence on team morale and contacts and access to various services. There are relationships. It is something we feel very proud of lots of deserving charities and causes out there but as a practice.” this one is about people-power and that’s inspiring.”

10 Sanfilippo Children’s Foundation Our year in review - 2015 11 TELEVISION MEDIA COVERAGE MEDIA COVERAGE

OUR MEDIA SOCIAL MEDIA Thank you to the following bloggers, Our online blogging community and online journalists and fashion COVERAGE social media gems e-retailers for their support.

TV, newspaper, magazine PRESS COVERAGE Social media is an increasing means of @Retro Mummy Sheree (@captainand & radio coverage in 2015 promoting awareness and raising funds @ShowandTell thegypsykid) for the SCF. Our presence on Facebook @Wheels of Laughter Carla (@stylingthetribe) Media coverage of rare disease @BellaMumma Andrea Rembeck in particular has enjoyed marked growth (@tutudumonde) remains a challenge but the @Waystoamaze for an organisation our size. Engagement @sarinasurianojewels Sanfilippo Children’s Foundation @Kendrascatchycorner levels sit well above the industry average @stuffmumslike has gained significant exposure @kidsfashionblogger at 11% and this year we exceeded 5,200 @petitparisians and continues to make headlines, Keria & Karianne likes. Total reach is on average 10K people @bloggingmika allowing us to effectively educate (@freeandwildchild) but peaked at 40K people during our song @abeachcottage the Australian community about Pete & Harlow launch and the associated Hope for Isla (@thedaddyfashionstylist) @champagnecartel our cause. Coverage has largely and Jude campaign. Jodie (@bondi_mumma) Kristy Withers (@ focused on our innovative The Dick Smith Breakthrough for Charity initiative Claire (@clairecollected) incyinteriors) fundraising events and both was an opportunity to really engage with our online Miss Maya Photography @luluandmilly corporate and community community, as our loyal supporters took action and (@pics_missmaya) @dimplesandwhispers embraced the collaborative spirit of the social activities. Renata (@curatedby) media campaign, taking and submitting more than @rourkeandhenry 2,500 selfies. Marcia Leone @havealaughonme The SCF was profiled across all mainstream (@notsomumsy) media channels this year including 35 @uneekkidz The online blogging community has also got behind Sandy Zeccardi print newspaper articles, 7 magazine and the Sanfilippo cause, allowing the SCF to utilise this (@littlezkid) @jaime_king e-zine articles, 5 TV segments, and 12 radio powerful means of communication. An online auction Kerri Sackville @littlegatherer interviews. The majority of coverage was of designer children’s clothing hosted by IG (Instagram) Rebecca Sparrow @saplingchild generated by the release of the single This fashion blogger Carla Rodan from @Stylingthetribe @theinteriorsaddict Woman’s Work and the Sanfilippo 1000 event was a hit, helping us to reach a larger and wider Sara Knowles which captured media interest in regional NSW international audience. (@theinstashopper) @christieblizzard and Victoria.

Other highlights include a radio interview with Alan Jones, articles in two broadsheet dailies The Sydney Morning Herald and The RADIO COVERAGE Australian, as well segments on Channel 9’s Mornings and Today Show, Chanel 9 News Melbourne and Channel 7’s Morning Show.

Thanks to Ooh! Media, SCF has enjoyed pro- bono digital advertising at select shopping centres, cafés and restaurants, for two years in a row. This generated a ‘SPOTTO’ campaign in which our supporters photographed our advertisement in public spaces and shared across social media.

12 Sanfilippo Children’s Foundation Our year in review - 2015 13 A message SPECIAL THANKS from our

The Sanfilippo Children’s Foundation would like to especially Founder acknowledge some exceptional gems

GRANT GIVERS

The Foundation has been a proud recipient of a grant from these generous foundations and organisations:

We talk a lot about the notion of people-powered medicine and we are seeing it Count Foundation Hacket Foundation in practise at the Sanfilippo Children’s Foundation. Honda Foundation Ripple Foundation It’s been a year packed with activity and one in which great progress has been made. The foundations of our first project are now firmly in place. We have partnered with a US-based biotech (Abeona Therapeutics) which is running a gene therapy program and engaged an Australian clinical team to establish a trial site on Australian soil that can run this program in 2016.

PRO-BONO SUPPLIERS For the first time kids with Sanfilippo and their families have the smallest glimmer of hope that they might These suppliers have provided much-valued ongoing time, material and resources to our cause: face a different future than the one that they thought they were. It means Australian children will receive treatment, a shot at life. And overall science will be that one step further towards an eventual cure.

The work of our Foundation is carried out by a dedicated board, a small but passionate team and a Accountants CXC Lawyers Clifford Chance growing group of committed volunteers, from which we have enjoyed the most extraordinary support (Jane Ann Gray) and generosity. This year in particular we have learned that individual everyday people really can make a Advertising Ooh! Media Photographers Little Love Photography difference and make things happen. Auditors Bentleys Miss Maya Pics Hayley Grace Photography Whilst we aren’t there yet, thanks to the incredible support of our strategic partners, corporate donors Graphic Design Katrina Commyns Sam Elsom and our extraordinary community, we will get there ...we will get a treatment for kids battling Sanfilippo Jacqui Greig (Captain and the Gypsy Kid) today and those born with it tomorrow. Noleen Lance Heartfelt May Leong We are full of hope for the year ahead. 2016 looks as if it will be as busy and fruitful as the past year Danielle O’Brien Art Alex Moller has been. We are excited by what we can achieve together! Carlma Photography Harry Morgan Kevin O’Sullivan With gratitude, Jacinta Weir Printers AAP Print KwikKopy St Leonards Lindfield Print & Copy Centre Event Production Jo Bilous Print Junction Grant Writing Amy McCudden Megan Donnell Public Relations Connect PR Media Treehouse Creative Founder of the Sanfilippo Children’s Foundation

14 Sanfilippo Children’s Foundation Our year in review - 2015 15 ABOUT THE TEAM MEET THE TEAM SCF BOARD MEMBERS

The Sanfilippo Children’s Team are a dedicated group of individuals SANFILIPPO CHILDREN’S

Professor John Hopwood AM FAA FOUNDATION SCIENTIFIC ADVISOR AMBASSADORS Sébastien Izambard Angeline Veeneman Singer from pop opera Professor John Hopwood recently group Il Divo retired as Director of the Lysosomal PATIENT INFORMATION & PROGRAMS Diseases Research Unit (LDRU). He Angeline's focus is on building our knowledge was previously at the Women’s and about the disease and associated medical Children’s Hospital, then moved to research, and aligning our research strategy accordingly. the South Australian Health and Medical Research Institute Megan Donnell (SAHMRI), Adelaide, Australia. The Unit is world-renowned FOUNDER & DIRECTOR for its research into the diagnosis, treatment and biology of lysosomal diseases including the Mucopolysaccharidoses Megan is responsible for providing (MPS) group of metabolic disorders. strategic vision, leadership and development of the Foundation within Professor Hopwood has dedicated four decades of research the direction set by the Board of Jessica McNamee Sonia Marinelli into genetically inherited disorders that affect children Directors. Megan is the Foundation’s Australian film & TV news reporter, including Sanfilippo Syndrome. A man of the people, key liaison between the Foundation, TV actress Channel 9 Professor Hopwood is particularly passionate about meeting partner organisations, funding groups, families and patients living with MPS and learning about their associated research and regulatory day-to-day challenges with the disease. bodies and patient groups globally. Daniel Madhavan INVESTMENTS & NETWORKS Daniel’s key activities include engaging with support networks to promote foundation awareness and increasing the scale of Foundation investment.

Dan Illic Catherine Bouchier Comedian & radio host Soprano opera singer

Ingrid Maack Nicole Fasseau Erene Keriakos COMMUNICATIONS & FUNDRAISING OPERATIONS MANAGER (PART- BOOKKEEPER PART-TIME MANAGER (PART-TIME) TIME) Erene works one day Ingrid is our primary communications Nicole is responsible per week providing Mark Arnold liaison. She is responsible for social for general operations, bookkeeping services STRATEGY & GOVERNANCE media and external communications administration, relationship for the Foundation. Mark is responsible for strategy and governance in line with the Foundation’s mission. management, corporate of the Foundation. His key activities include In addition, Ingrid manages our programs and broadening defining organisational strategy to achieve the community fundraising events the Foundation’s fundraising mission and objectives of the foundation and Jeannette Rowe Silas Moss and activities. opportunities. establishing processes to facilitate compliance Children’s book author Sports journalist and blogger with governance and regulatory requirements.

16 Sanfilippo Children’s Foundation Our year in review - 2015 17 OUR FAMILY STORIES

Every Family has a story. These are just some of the Australian children battling Sanfilippo Syndrome.

Jayda no longer talks or sings Jacob is a gorgeous little Skye likes dress-ups, AFL Isla and Jude are brother and sister. They like she once did. Her little man with a heart of gold. and playing with big brother We sadly say goodbye adore each other. Isla loves dressing up and sister Lucy sometimes calls He is an affectionate boy, Luke. She has a bubbly and singing. Jude is full of boundless energy In 2015 we lost a much-loved Australian Jayda her “baby”. Her parents who loves his little sister vibrant personality and is and makes friends wherever he goes. It is child to Sanfilippo Syndrome. Dylan Jessop like to watch old videos of Alexis. Without treatment, always up to mischief. Her lost his battle in July. Dylan was just 12 years Jayda to remember the way one day Jacob will break family fear one day she will unimaginable one may lose not only their old but touched the hearts of everyone he she was. hearts but not in the way his lose her sense of adventure. sibling but also their best friend. grandmother expected. encountered in his short but full life. To honour Dylan and his spirit, the Jessop family together with the Sanfilippo Children’s Foundation set up an ‘In Memoriam’ page, and asked for friends and family to make a donation in Dylan’s memory. This initiative generated $3,350 and donors also left messages of support for the Jessop family. About the experiences Dylan is sadly missed by his loving parents Peter is a happy little boy Kyuss can kick a ball, ride a Mikayla loves spending Meckenzie is a social of Australian families who likes to play with his bike and zoom on his scooter. time with her big brother butterfly and is full of who are impacted by Matt and Louise and younger brothers Sanfilippo Syndrome. little brother Eliya and sister He likes to ride his quad bike Jake, who sits and reads to personality and giggles. Ethan and Ayden. Natalie. Peter touches many on his grandparents’ farm. her. She is in a wheelchair Meckenzie’s big brother hearts. While he still walks, Kyuss has hearing loss but and eats through a tube. Kyle treasures each and Visit our website: RIP Dylan Jessop... gone too soon. Peter no longer speaks. Sanfilippo will steal much Her parents miss the every day he has left sanfilippo.org.au more than his hearing. sound of her voice. with his little sister.

18 Sanfilippo Children’s Foundation Our year in review - 2015 19 For a full copy of our audited report, please THE FINANCIALS contact us via email - [email protected] THE DISEASE FACT SHEET or by phoning 1800 664 878 What is Sanfilippo (MPSIII)?

FY14/15 FY13/14

INCOME DONATIONS $387,996 $371,825 FUNDRAISING ACTIVITIES $384,278 $121,736 NON-GOVERNMENT GRANTS $87,100 $15,000 INTEREST INCOME $13,970 $2,248 MERCHANDISE $6,795 $912 TOTAL INCOME $880,139 $511,721

FUNDRAISING COSTS $106,940 $42,312

OPERATING EXPENSES ADMINISTRATION $56,191 $10,223 MARKETING $17,694 $564 PROGRAMS $21,781 $3,402 TOTAL OPERATING EXPENSES $95,668 $14,189

NET PROFIT (FOR DISTRIBUTION) $677,531 $455,220

$1,200,000

$1,100,000

$1,000,000

$900,000 $677,531 COMMITTED TO CLINICAL $800,000 RESEARCH FY14/15 DIRECT PEER 2 PEER $700,000 DONATIONS

$600,000

$500,000 EVENTS $400,000

GRANTS $300,000 $455,221 OTHER $200,000 FY13/14 INVESTED IN CLINICAL RESEARCH Fundraising sources (FY14/15)

$100,000

DONATIONS

DONATIONS DIRECT DIRECT DIRECT DIRECT The disease does not yet have a cure and only limited pallative treatment NET PROFIT DISTRIBUTION currently exist; average life expectancy is 15-20 years. However medical research has recently achieved promising break-throughs with real hopes for the future.

20 Sanfilippo Children’s Foundation Our year in review - 2015 21

EXAMPLE

DONATIONS DIRECT DIRECT GRANTS THE RESEARCH OUR RESEARCH PARTNERSHIPS About our Research Partnership with Abeona Therapeutics

The last year has been very active from a research perspective and brings renewed In January 2015, the Sanfilippo Children’s Foundation finalised a legal agreement and hope for the whole Sanfilippo community across the world. With multiple programs in executed its first scheduled contribution of USD$200,000 (AUD$236,000) to help fund early clinical trial stages, there are increasing opportunities for children with Sanfilippo Abeona’s international gene therapy clinical trial for children affected by Sanfilippo to access experimental treatments that have a real possibility to change their lives and Syndrome. In total, the Foundation will contribute a total of USD$1 million (est. AUD$1.3 that of their families. million) towards the funding of Abeona’s programs. Contributions are aligned to agreed As a monogenic disease that affects the Central clinic, there is also a lot of preclinical research being milestones being achieved and include the establishment of a clinical trial site in Nervous System, Sanfilippo Syndrome is a condition carried out to explore alternative ways and treatments Australia in 2016. that it is considered as one from which scientists, for Sanfilippo Syndrome. In November 2015, Abeona executives including Tim How does Abeona’s gene therapy treatment work? researchers and biopharmaceutical groups can learn. Miller, CEO and Michelle Berg, Vice President Patient Gene therapy is the use of DNA as a potential therapy Finding an effective treatment for Sanfilippo creates Research activities, especially at clinical trial stage, have Advocacy travelled to Australia. The purpose of the to treat a disease. In many disorders, particularly application opportunities for other more complex historically been largely driven by North American and visit was to engage a clinical team here in Australia, genetic diseases caused by a single genetic defect, genetic and neurological disorders. European research institutions and biopharmaceutical companies. We intend to change this! These groups perform due diligence on the preferred clinical trial site gene therapy aims to treat a disease by delivering the Gene Therapy and Enzyme Replacement Therapy recognise the value and need to expand their research and to provide a full briefing to Australian families. correct copy of a faulty gene into a patient’s cells. The remain the principal therapeutic avenues pursued by and trials to other countries such as Australia, with healthy, functional copy of the therapeutic gene then As stated on the Abeona website, Abeona research groups. Due to their higher level of incidence, several major programs now evaluating Australian helps the cell function correctly. In gene therapy, DNA Therapeutics is focused on developing and delivering MPSIII Types A and B are the types where research is institutions as potential sites for international clinical that encodes a therapeutic protein is packaged within a gene therapy and plasma-based products for severe most advanced. In addition to programs entering the trial execution. “vector”, often a “naked” virus, which is used to transfer and life-threatening rare diseases. the DNA to the inside of cells within the body. Gene Developing therapies for rare disease requires new therapy can be delivered by a direct injection, either RESEARCH GROUP TYPE STAGE TRIALS TIMEFRAME PLANNED TRIAL SITES approaches and strong collaboration between intravenously (IV) or directly into a specific tissue in Gene Therapy researchers, industry, regulators and patient groups. the body, where it is taken up by individual cells. Once Completed in 2015 Institut Pasteur & Uniqure B Phase l/ll Europe (France) Abeona was forged from the company’s close inside cells, the correct DNA becomes expressed (results pending) collaborations with key stakeholders all dedicated by the cell machinery, resulting in the production of Abeona Therapeutics & Commencing 2015 USA, Europe (Spain), A & B Phase l/ll to transforming new biotechnology insights into therapeutic protein, which in turn treats the patient’s Nationwide Children's Hospital / 2016 Australia breakthrough treatments for rare diseases. disease and can provide long-term benefit. Esteve & Autonomous A Phase l/ll Commencing 2016 Europe (Spain) University of Barcelona Lysogene A Phase lll Commencing 2016 Europe, USA 1. MPS III causes lysosomal 2. GAGs accumulate in the brain 3. Corrective DNA is inserted to enzyme deficiency resulting and body resulting in cognitive an AAV9 virus delivery vector University of Manchester & C Phase l/ll Commencing 2017 Europe in excessive accumulation of decline, motor dysfunction and which allows it to go into the H.A.N.D.S Consortium glycosaminoglycans (GAGs). premature death. central nervous system and Enzyme Replacement Therapy body tissues. Europe, Americas Shire A Phase llb In Progress (multiple countries) Alexion (formerly Synageva) B Phase l/ll In Progress USA, UK AAV Vector Australia, Brazil, Columbia, Corrective DNA Biomarin B Phase l/ll In Progress Europe (Germany, Turkey, Spain, UK), Taiwan Pre-clinical studies in the Substrate Reduction Therapy gene therapy programs have shown promising University of Manchester All Phase lll In Progress UK Virus taken results that the treatment (Genistein) Protein into cell via has the potential to safely expressed endosome Stem Cell Therapy halt disease progression in University of Manchester C Phase l/ll Start 2016 UK children. No safety issues DNA forms have been identified in episome treated MPSIII animals and there has been no evidence ... AND MUCH MORE YET TO COME. Whilst science is progressing well, providing access to treatments for for autoimmune responses. Endosome Virus binds Australian children with Sanfilippo remains a major challenge, particularly given the inherent uncertainty linked to breaks 4. ABO-101 / AVO-102 (AAV9) to nucleus & down (Source: Abeona clinical trials and their progress path. Whilst the prospect of clinical trials coming imminently to Australia represents 6. Corrected lysosomal releases DNA administered in single Therapeutics) an incredible opportunity for the Australian patients community, the Sanfilippo Children’s Foundation needs to degradation results intravenous (iv) infusion. ABO- 101 is for MPS IIIB and continue its efforts to invest in and support a range of research programs and activities. in small diffusable 5. ABO-101 / ABO-102 delivers corrective gene end products. to the central nervous system and body. ABO- 102 is for MPS IIIA.

22 Sanfilippo Children’s Foundation Our year in review - 2015 23 FUNDING RESEARCH, FINDING GEMS Our logo supports our mission to fund and drive research, leading to effective treatments for our ABOUT OUR LOGO Sanfilippo children. Along the Rainbow double helix symbol: The way we look for the gems - those rainbow is a universally recognised treasured moments our Sanfilippo symbol for hope and the double families share with their children helix represents genetics, which whilst discovering true gems, our underlie this disease, and science supporters assisting in our journey upon which our hope for effective as we all continue the search for the treatment is based. ultimate gem - a cure.

Thank you to our gems - our valued supporters, donors, sponsors, suppliers, volunteers and fundraisers. We couldn’t do what we do at the SCF without you!

Contact

Sanfilippo Children’s Foundation PO Box 475 Freshwater, NSW 2096, Australia

E [email protected] T 1800 664 878

This Year in Review document has been produced by professionals who have discounted and/or donated their time and services. We would like to thank designer Alex Moller and Lindfield Print and Copy Centre.

24 Sanfilippo Children’s Foundation